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Efficacy and Safety of Obeticholic Acid in the Treatment of Primary Biliary Cholangitis

Primary Purpose

Primary Biliary Cholangitis

Status

Recruiting

Phase

Phase 3

Locations

China

Study Type

Interventional

Intervention

Obeticholic Acid Tablets（OCA）

UDCA

Placebo

About this trial

This is an interventional treatment trial for Primary Biliary Cholangitis

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Age ≥18 and ≤75 years；
Definite PBC diagnosis, as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors: ① Indicators reflecting cholestasis such as elevated ALP；② Positive antimitochondrial antibody (AMA) or AMA-M2, or positive PBC-specific antibody (anti-GP210 and/or anti-SP100) if AMA negative；③ Liver biopsy consistent with PBC；
At least 1 of the following qualifying biochemistry values: ① ALP ≥ 1.67x ULN ；② Total bilirubin > ULN but < 2x ULN；
Taking UDCA for at least 6 months (stable dose for ≥ 3 months) prior to Day 0, or unable to tolerate UDCA (no UDCA for ≥ 3 months) prior to Day 0；
Understand the study, comply with the study protocol, and voluntarily sign the informed consent form.

Exclusion Criteria:

Patients who took obeticholic acid within 3 months prior to Day 0;
Known hypersensitivity to obeticholic acid, ursodeoxycholic acid;
History or presence of other concomitant liver diseases;
Cirrhosis-related complications or end-stage liver disease manifestations;
Serum creatinine (Cr) ≥ 1.5 × ULN and serum creatinine clearance < 60 mL/min;
Patients with severe pruritus or requiring systemic drug therapy within 2 months prior to Day 0;
Patients with HIV or syphilis infection;
Presence of diseases or physiological conditions that interfere with the absorption, distribution, metabolism or excretion of test drugs, such as inflammatory bowel disease and previous gastric bypass surgery;
Presence of diseases that may cause non-hepatogenic ALP elevation, or diseases that may lead to a life expectancy of less than 2 years;
Administration of the following drugs within 6 months prior to Day 0: azathioprine, colchicine, cyclosporine, methotrexate, mycophenolate mofetil, pentoxifylline; fenofibrate or other fibrates; budesonide and other systemic corticosteroids; hepatotoxic drugs (including α-methyldopa, sodium valproate, isoniazid, nitrofurantoin, etc.);
Administration of the following drugs within 12 months prior to Day 0: antibodies or immunotherapy against interleukins or other cytokines or chemokines;
Patients with serious cardiovascular system, digestive system, respiratory system, urinary system, nervous system, mental illness, immunodeficiency disease, and judge by investigators that they are not suitable for participating in the trial;
Other conditions that are not considered appropriate by the investigator.

Sites / Locations

Beijing Friendship HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

OCA 5 mg titrated to 10 mg ± UDCA

Placebo ± UDCA

Arm Description

OCA 5 mg once daily for 3 months and then titrating up to 10 mg based on tolerability and response. Subjects receiving UDCA continued taking UDCA throughout the trial. If the subjects could not tolerate UDCA, they were not treated with UDCA.

Placebo once daily. Subjects receiving UDCA continued taking UDCA throughout the trial. If the subjects could not tolerate UDCA, they were not treated with UDCA.

Outcomes

Primary Outcome Measures

Percentage of PBC patients reaching the compound endpoint after 12 months of treatment (Compound endpoint: alkaline phosphatase (ALP) < 1.67× Upper Limit of Normal(ULN), and ALP decrease ≥ 15% from baseline, and total bilirubin ≤ ULN )

Compound endpoint: ALP < 1.67× ULN, and ALP decrease ≥ 15% from baseline, and total bilirubin ≤ ULN

Secondary Outcome Measures

Absolute change and percentage change of ALP, total bilirubin, direct bilirubin, ALT, AST and GGT from baseline to Month 3, 6, 9 and 12

Blood samples were evaluated for ALP, total bilirubin, direct bilirubin, ALT, AST and GGT levels. Absolute change and percentage change of ALP, total bilirubin, direct bilirubin, ALT, AST and GGT from baseline to Month 3, 6, 9 and 12 are presented.

Quality of life for PBC measure (PBC-40) score percentage change from baseline to Month 3, 6, 9 and 12

PBC-40 score was evaluated at certain visits. PBC-40 score percentage change from baseline to Month 3, 6, 9 and 12 is presented.

Full Information

NCT ID

NCT05450887

First Posted

July 4, 2022

Last Updated

September 13, 2023

Sponsor

Nanjing Chia-tai Tianqing Pharmaceutical

1. Study Identification

Unique Protocol Identification Number

NCT05450887

Brief Title

Efficacy and Safety of Obeticholic Acid in the Treatment of Primary Biliary Cholangitis

Official Title

A Randomized, Double-blind, Multicenter, Placebo-controlled Phase III Clinical Trial to Evaluate the Efficacy and Safety of Obeticholic Acid in Patients With Primary Biliary Cholangitis

Study Type

Interventional

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Recruiting

Study Start Date

September 23, 2021 (Actual)

Primary Completion Date

June 2024 (Anticipated)

Study Completion Date

June 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Nanjing Chia-tai Tianqing Pharmaceutical

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

Obecholic acid is a modified bile acid and Farnesoid X receptor (FXR) agonist. FXR is a key regulator of bile acid synthesis and transport. Bile acids are used by the body to help with digestion. Conventional therapy with obecholic acid will improve liver function of patients with (primary biliary cholangitis)PBC. The main objectives of the study were to assess the effects of Obeticholic Acid (OCA) on serum alkaline phosphatase (ALP) and total bilirubin, together as a composite endpoint and on safety in participants with PBC.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Primary Biliary Cholangitis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

156 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

OCA 5 mg titrated to 10 mg ± UDCA

Arm Type

Experimental

Arm Description

Arm Title

Placebo ± UDCA

Arm Type

Placebo Comparator

Arm Description

Placebo once daily. Subjects receiving UDCA continued taking UDCA throughout the trial. If the subjects could not tolerate UDCA, they were not treated with UDCA.

Intervention Type

Drug

Intervention Name(s)

Obeticholic Acid Tablets（OCA）

Intervention Description

Obeticholic Acid：Once a day (QD) by mouth (PO).

Intervention Type

Drug

Intervention Name(s)

UDCA

Intervention Description

UDCA：13~15 mg/kg/day

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo：Once a day (QD) by mouth (PO).

Primary Outcome Measure Information:

Title

Description

Compound endpoint: ALP < 1.67× ULN, and ALP decrease ≥ 15% from baseline, and total bilirubin ≤ ULN

Time Frame

up to 12 months

Secondary Outcome Measure Information:

Title

Absolute change and percentage change of ALP, total bilirubin, direct bilirubin, ALT, AST and GGT from baseline to Month 3, 6, 9 and 12

Description

Time Frame

up to 12 months

Title

Quality of life for PBC measure (PBC-40) score percentage change from baseline to Month 3, 6, 9 and 12

Description

PBC-40 score was evaluated at certain visits. PBC-40 score percentage change from baseline to Month 3, 6, 9 and 12 is presented.

Time Frame

up to 12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Age ≥18 and ≤75 years； Definite PBC diagnosis, as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors: ① Indicators reflecting cholestasis such as elevated ALP；② Positive antimitochondrial antibody (AMA) or AMA-M2, or positive PBC-specific antibody (anti-GP210 and/or anti-SP100) if AMA negative；③ Liver biopsy consistent with PBC； At least 1 of the following qualifying biochemistry values: ① ALP ≥ 1.67x ULN ；② Total bilirubin > ULN but < 2x ULN； Taking UDCA for at least 6 months (stable dose for ≥ 3 months) prior to Day 0, or unable to tolerate UDCA (no UDCA for ≥ 3 months) prior to Day 0； Understand the study, comply with the study protocol, and voluntarily sign the informed consent form. Exclusion Criteria: Patients who took obeticholic acid within 3 months prior to Day 0; Known hypersensitivity to obeticholic acid, ursodeoxycholic acid; History or presence of other concomitant liver diseases; Cirrhosis-related complications or end-stage liver disease manifestations; Serum creatinine (Cr) ≥ 1.5 × ULN and serum creatinine clearance < 60 mL/min; Patients with severe pruritus or requiring systemic drug therapy within 2 months prior to Day 0; Patients with HIV or syphilis infection; Presence of diseases or physiological conditions that interfere with the absorption, distribution, metabolism or excretion of test drugs, such as inflammatory bowel disease and previous gastric bypass surgery; Presence of diseases that may cause non-hepatogenic ALP elevation, or diseases that may lead to a life expectancy of less than 2 years; Administration of the following drugs within 6 months prior to Day 0: azathioprine, colchicine, cyclosporine, methotrexate, mycophenolate mofetil, pentoxifylline; fenofibrate or other fibrates; budesonide and other systemic corticosteroids; hepatotoxic drugs (including α-methyldopa, sodium valproate, isoniazid, nitrofurantoin, etc.); Administration of the following drugs within 12 months prior to Day 0: antibodies or immunotherapy against interleukins or other cytokines or chemokines; Patients with serious cardiovascular system, digestive system, respiratory system, urinary system, nervous system, mental illness, immunodeficiency disease, and judge by investigators that they are not suitable for participating in the trial; Other conditions that are not considered appropriate by the investigator.

Facility Information:

Facility Name

Beijing Friendship Hospital

City

Beijing

ZIP/Postal Code

100050

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

ChunXiu Yang

Phone

010-63138628

chunxiuyang@sina.com

First Name & Middle Initial & Last Name & Degree

JiDong Jia

First Name & Middle Initial & Last Name & Degree

Hong You

12. IPD Sharing Statement

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Efficacy and Safety of Obeticholic Acid in the Treatment of Primary Biliary Cholangitis

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